National Center for Advancing Translational Sciences (NIH), USA

2017

  • AN ENDOPLASMIC RETICULUM CALCIUM STABILIZER FOR THE TREATMENT OF WOLFRAM SYNDROME(website)
  • AN ORAL ULTRA LONG-ACTING IVERMECTIN FOR MALARIA ELIMINATION(website)
  • CONFERENCE ON CLINICAL RESEARCH FOR RARE DISEASES (CCRRD)(website)
  • DRUG DEVELOPMENT FOR TUBEROUS SCLEROSIS COMPLEX AND OTHER PEDIATRIC EPILEPTOGENIC DISEASES USING NEUROVASCULAR AND CARDIAC MICROPHYSIOLOGICAL MODELS(website)
  • ENHANCING PATIENT ENGAGEMENT IN NEUROFIBROMATOSIS CLINICAL TRIALS(website)
  • GENE THERAPY PLATFORM FOR RARE DISEASES(website)
  • IDENTIFICATION OF ACTIVATORS OF PMP22 EXPRESSION FOR TREATMENT OF CMT1A(website)
  • IDENTIFICATION OF INHIBITORS OF ZIKA PROTEASE (NS2B-NS3)(website)
  • IDENTIFICATION OF NOVEL ANTIMARIAL DRUGS TARGETING ASEXUAL BLOOD STAGE OF P. VIVAX(website)
  • IDENTIFICATION OF SMALL MOLECULE ER MODULATORS FOR WOLFRAM SYNDROME(website)
  • IN VIVO EDITING FOR HEMOPHILIA GENE THERAPY(website)
  • JUVENILE MYOSITIS(website)
  • METARRESTIN FOR THE TREATMENT OF PANCREATIC CANCER(website)
  • NOVEL NANOPARTICLE-BASED ENZYME REPLACEMENT THERAPY FOR HUNTER SYNDROME(website)
  • P97 INHIBITORS FOR TREATMENT OF IBMPFD/ALS(website)
  • PERSONALIZED NEURAL STEM CELL THERAPY FOR CANCER(website)
  • PRECLINICAL EVALUATION OF VORINOSTAT IN ALOPECIA AREATA(website)
  • PRKACA FOR FIBROLAMELLAR HEPATOCELLULAR CARCINOMA(website)
  • QHTS TO IDENTIFY COMPOUNDS AGAINST MERKEL CELL CARCINOMA (MCC)(website)
  • QHTS TO IDENTIFY INHIBITORS OF ZIKA VIRUS(website)
  • QUANTITATIVE SYSTEMS PHARMACOLOGY APPROACH TO IDENTIFY CYTOPROTECTIVE AGENTS AND PATHWAYS FOR HUNTINGTON’S DISEASE(website)
  • RARE AND UNDIAGNOSED DISEASES: DISCOVERY AND MODELS OF PRECISION THERAPY(website)
  • RECONSTITUTED HIGH DENSITY LIPOPROTEIN PARTICLES AS SIRNA CARRIERS(website)
  • SOFTWARE PLATFORM TO STRATIFY PATIENTS FOR TREATMENT ARM RANDOMIZATION IN HUMAN CLINICAL TRIALS USING PATIENT-LEVEL PREDICTIVE MODELS(website)
  • SUBCUTANEOUS REPLACEMENT ENZYMES FOR FABRY DISEASE(website)
  • TARGETING GLUCOSE METABOLISM FOR THE TREATMENT OF HEPATOCELLULAR CARCINOMA(website)
  • USING THE PREIMPLANTATION FACTOR (PIF) TO TREAT GRAFT-VERSUS-HOST DISEASE(website)
  • UTILIZATION OF PHENOTYPIC PRECISION MEDICINE TO IDENTIFY OPTIMAL DRUG COMBINATIONS FOR THE TREATMENT OF HEPATOCELLULAR CARCINOMA(website)

2016

  • 3D BIOPRINTING OF HUMAN NATIVE-LIKE TISSUES AS DISEASE-IN-A-DISH MODELS FOR DRUG DISCOVERY(website)
  • A DRUG BASED APPROACH FOR INTEGRIN-MEDIATED ALLEVIATION FOR MUSCULAR DYSTROPHY(website)
  • A DRUG-SCREENING PLATFORM FOR AUTISM SPECTRUM DISORDERS USING HUMAN NEURONS AND ASTROCYTES(website)
  • A NOVEL COMPOUND FOR TARGETED TREATMENT OF CBF LEUKEMIA(website)
  • A PROTEIN REPLACEMENT DRUG FOR FRIEDREICHS ATAXIA(website)
  • ALPHA-1 ANTITRYPSIN (AAT) DEFICIENCY(website)
  • BIOMEDICAL DATA TRANSLATOR TECHNICAL FEASIBILITY ASSESSMENT AND ARCHITECTURE DESIGN(website)
  • CONFERENCE ON CLINICAL RESEARCH FOR RARE DISEASES (CCRRD)(website)
  • CTSA SUPPLEMENT(website)
  • CYCLODEXTRIN FOR NIEMANN-PICK TYPE C1 DISEASE(website)
  • DEVELOPMENT OF BONE MORPHOGENETIC PROTEIN INHIBITORS TO TREAT BLOOD AND BONE DISORDERS(website)
  • DEVELOPMENT OF MALARIA TRANSMISSION-BLOCKING DRUGS(website)
  • DEVELOPMENT OF NEUROSTEROIDS FOR LYSOSOMAL STORAGE DISORDERS(website)
  • DEVELOPMENT OF NOVEL ANTIMALARIAL TARGETS AND COMBINATION THERAPIES TO ADDRESS DRUG RESISTANCE(website)
  • DEVELOPMENT OF NUCLEIC ACID DELIVERY PLATFORM BASED ON POLYMERIC CXCR4 ANTAGONISTS(website)
  • DEVELOPMENT OF PLATELET TRANSCRIPTOME SIGNATURES IN PATIENTS WITH MYELOPROLIFERATIVE NEOPLASMS(website)
  • DEX-M74 FOR GNE MYOPATHY(website)
  • DISEASE-SPECIFIC INTEGRATED MICROPHYSIOLOGICAL HUMAN TISSUE MODELS(website)
  • EARLY CHECK: A COLLABORATIVE INNOVATION TO FACILITATE PRE-SYMPTOMATIC CLINICAL TRIALS IN NEWBORNS(website)
  • FOURTH INTERNATIONAL MEDICAL SYMPOSIUM ON ERDHEIM-CHESTER DISEASE(website)
  • GLYCOGEN STORAGE DISEASE III ENGINEERED HUMAN SKELETAL MUSCLE RARE DISEASE MODEL(website)
  • HIGH CONTENT SCREENING USING 3-D MODELS OF OVARIAN CANCER(website)
  • HTS ENABLED EXAMINATION OF DRUG COMBINATIONS(website)
  • HUMAN DNA TRANSLESION SYNTHESIS POLYMERASE KAPPA(website)
  • IDENTIFICATION AND VALIDATION OF TARGETS FOR THERAPEUTIC INTERVENTION IN RARE DISEASES OF INTERMEDIARY METABOLISM(website)
  • IDENTIFICATION OF GPR52 ANTAGONISTS AS POTENTIAL TREATMENTS FOR HUNTINGTON DISEASE(website)
  • IDENTIFICATION OF INHIBITORS OF PLASMODIUM FALCIPARUM HEXOKINASE (PFHK)(website)
  • IDENTIFICATION OF INHIBITORS OF THE N370S MUTANT FORM OF GLUCOCEREBROSIDASE AS A POTENTIAL THERAPY FOR GAUCHER DISEASE(website)
  • IDENTIFICATION OF INHIBITORS OF YES1 KINASE ACTIVITY AND DETERMINATION OF YES1 POLYPHARMACOLOGY OF KINASE INHIBITORS(website)
  • IDENTIFICATION OF SMALL MOLECULE INHIBITORS OF PHF5A FOR GLIOBLASTOMA(website)
  • IDENTIFICATION OF SMALL MOLECULE THAT ACT ON GSP; THE ETIOLOGIC MUTATION RESPONSIBLE FOR FIBROUS DYSPLASIA/MCCUNE-ALBRIGHT SYNDROME(website)
  • IDENTIFICATION OF SMALL MOLECULES THAT DECREASE THE ACTIVITY OF THE HUNTINGTIN GENE PROMOTER FOR THE POTENTIAL TREATMENT OF HUNTINGTON DISEASE(website)
  • INHALED GM-CSF THERAPY OF AUTOIMMUNE PULMONARY ALVEOLAR PROTEINOSIS(website)
  • INHIBITION OF AMA1-RON2 INTERACTION TO DISRUPT INVASION OF MALARIA PARASITE(website)
  • IPGLYCERMIDES NOVEL POTENT AND SELECTIVE INHIBITORS OF PARASITIC PHOSPHOGLYCERATE MUTASE(website)
  • KETOROLAC AND RELATED NSAIDS FOR TARGETING RHO-FAMILY GTPASES IN OVARIAN CANCER(website)
  • LONG-ACTING PARATHYROID HORMONE ANALOG FOR THE TREATMENT OF HYPOPARATHYROIDISM(website)
  • LUM-001 AS A TREATMENT FOR CREATINE TRANSPORTER DEFICIENCY(website)
  • MODELING A RARE AUTONOMIC DISEASE(website)
  • MODELING METABOLIC INTERACTIONS OF LIVER AND FAT WITH INTEGRATED MICROPHYSIOLOGICAL SYSTEMS(website)
  • MODELLING RETT SYNDROME IN VASCULARIZED NEURAL ASSEMBLIES(website)
  • NPC/MIPE TOXICITY SCREEN OF IDH1 INHIBITORS AGAINST CHOLANGIOCARCINOMA CELL LINES(website)
  • PEROXISOME BIOGENESIS DISORDERS (PBDS)(website)
  • PHENOTYPIC ASSAY DESIGN AND DEVELOPMENT FOR RARE AND NEGLECTED DISEASES(website)
  • PHENOTYPIC TOXICITY SCREENING IN FAK/EWINGS SARCOMA(website)
  • PORTABLE IRIDIUM-ELECTRODE INHALED NITRIC OXIDE GENERATOR FOR TREATMENT OF LUNG DISEASES(website)
  • POTENTIAL THERAPEUTICS AGAINST ORTHOPOXVIRUSES(website)
  • QHTS TO IDENTIFY ACTIVATORS OF E47(website)
  • QHTS TO IDENTIFY INHIBITORS OF DYT1 INCLUSION FORMATION(website)
  • QHTS TO IDENTIFY INHIBITORS OF NNMT1(website)
  • QUANTUM MODEL REPURPOSING OF CETHROMYCIN FOR LIVER STAGE MALARIA(website)
  • REDUCTION OF ATXN2 EXPRESSION AS A MEANS TO TREAT SPINOCEREBELLAR ATAXIA TYPE 2 (SCA2)(website)
  • REPURPOSING AN EU THERAPEUTIC FOR HEMOGLOBINOPATHIES(website)
  • REPURPOSING PYRONARIDINE AS A TREATMENT FOR THE EBOLA VIRUS(website)
  • STUDIES OF TUMOR-PENETRATING MICROPARTICLES FOR PANCREATIC CANCER TRAUMA(website)
  • THE DEVELOPMENT OF MULTIKINASE INHIBITORS FOR THE TREATMENT OF SELECTED CANCERS(website)
  • TOXICOLOGY IN THE 21ST CENTURY PROGRAM (TOX21) – GENOMIC TOXICOLOGY(website)
  • TREATMENT OF ACID CERAMIDASE DEFICIENCY(website)
  • USE OF RAPAMYCIN FOR THE TREATMENT OF HYPERTROPHIC CARDIOMYOPATHY IN PATIENTS WITH LEOPARD SYNDROME(website)

2015

  • 4TH INTERNATIONAL RASOPATHIES SYMPOSIUM(website)
  • A TARGETED IMMUNOMODULATOR FOR THE TREATMENT OF ACUTE SICKLE CRISIS(website)
  • ALS CLINICAL TRIALS GUIDELINES
  • DYSTONIA COALITION(website)
  • EVALUATION OF A CATHEPSIN S INHIBITOR AS A POTENTIAL DRUG FOR CHAGAS DISEASE(website)
  • EVALUATION OF AZD9291 IN GLIOBLASTOMA PATIENTS WITH ACTIVATED EGFR(website)
  • EXPANSION OF AN EFFICIENT DRUG REPURPOSING PLATFORM FOR RARE GENETIC DISEASES(website)
  • FIBER DESIGN AND ASSESSMENT FOR DEVELOPMENT OF A NOVEL BIOMIMETIC MEDICAL DEVICE(website)
  • RAPID POINT-OF-CARE DIAGNOSTIC FOR EARLY DETECTION OF DENGUE INFECTION(website)
  • RAPID, NEAR-PATIENT TESTS TO MONITOR AND GUIDE THERAPY IN SICKLE CELL DISEASE PATIENTS
  • RARE DISEASE RESEARCH TRAINING PROGRAM
  • WEE1 AND HDAC INHIBITION IN RELAPSED/REFRACTORY AML(website)

2014

  • ADMINISTRATIVE SUPPLEMENT: EXTRACELLULAR NON-CODING RNA BIOMARKERS OF HEPATOCELLULAR CANCER(website)
  • BRITTLE BONE DISORDERS CONSORTIUM OF THE RARE DISEASE CLINICAL RESEARCH NETWORK
  • DEVELOPMENT OF AN IPSC-DERIVED HUMAN VASCULAR SYSTEM FOR DRUG DISCOVERY AND DEVEL(website)
  • FLUORESCENT RNA FOCI ASSAY FOR FXTAS DRUG DISCOVERY(website)
  • FLUORESCENT RNA FOCI ASSAY FOR FXTAS DRUG DISCOVERY (website)
  • HPV COMMUNICATION TO MICROENVIRONMENT VIA EXOSOMES(website)
  • MOLECULAR LIBRARIES PROBE PRODUCTION CENTERS NETWORK(website)
  • PHASE I, SBIR CONTRACT, N43TR-14-002?AN ASSAY TO MEASURE SURROGATE BIOMARKERS FOR DMD?
  • PRECLINICAL INNOVATION(website)
  • RARE DISEASES DATA MANAGEMENT AND COORDINATING CENTER(website)

2013

  • A NEW CTSA PARTNERSHIP TO TRANSLATE AN ORAL CANCER BIOMARKER FROM LAB TO CLINIC (website)
  • BIOCOMPUTATION ACROSS DISTRIBUTED PRIVATE DATASETS TO ENHANCE DRUG DISCOVERY(website)
  • CLINICAL UTILITY OF SALIVARY EXRNA BIOMARKERS FOR GASTRIC CANCER DETECTION(website)
  • COMB EXTERNAL BEAM RADIOTHERAPY WITH 153SM-EDTMP TO TREAT HIGH RISK OSTEOSARCOMA
  • DEVELOPMENT OF A BIOCHEMICAL DIAGNOSIS FOR CREUTZFELDT-JAKOB DISEASE (website)
  • ENZYME REPLACEMENT THERAPY FOR SANFILIPPO A LYSOSOMAL RARE DISEASE (website)
  • EXOSOME BASED THERAPEUTICS IN HUNTINGTON’S DISEASE(website)
  • EXRNA BIOMARKERS FOR HUMAN GLIOMA(website)
  • EXTRACELLULAR NON-CODING RNA BIOMARKERS OF HEPATOCELLULAR CANCER(website)
  • NOVEL EXTRA CELLULAR RNA-BASED COMBINATORIAL RNA INHIBITION THERAPY(website)
  • PANCREATIC CANCER:ASSAY FOR EARLY DETECTION(website)
  • PARTNERING TO TREAT AN ORPHAN DISEASE DUCHENNE MUSCULAR DYSTROPHY(website)
  • PARTNERING TO TREAT AN ORPHAN DISEASE DUCHENNE MUSCULAR DYSTROPHY (website)
  • PHARMACOLOGICAL SUPPRESSION OF ROD OPSIN AS THERAPY FOR RETINITIS PIGMENTOSA(website)
  • PRECLINICAL DEVELOPMENT OF NOVEL MEK1/2 INHIBITORS TO TREAT INHERITED CARDIOMYOPA(website)
  • TARGETED DELIVERY OF MICRORNA-LOADED MICROVESICLE FOR CANCER THERAPY(website)
  • THERAPEUTIC STRATEGY FOR LAM (LYMPHANGIOLEIOMYOMATOSIS)(website)

2012

  • A NEW CTSA PARTNERSHIP TO TRANSLATE AN ORAL CANCER BIOMARKER FROM LAB TO CLINIC(website)
  • ANGELMAN SYNDROME (AS) (website)
  • ASSESSING THE IMPACT OF OSTEOGENESIS IMPERFECTA ON NON-SKELETAL SYSTEMS (website)
  • DRUG REPURPOSING AND REDISCOVERY FOR GASTROINTESTINAL STROMAL TUMORS(website)
  • EARLY DETECTION OF PANCREATIC CANCER USING ICE COLD-PCR(website)
  • MODELING OXIDATIVE STRESS AND DNA DAMAGE USING GI ORGANOTYPIC CULTURE SYSTEMS(website)
  • ONLINE PATIENT SELF-ASSESSMENT SYSTEM FOR CARE AND RESEARCH OF JOINT AND SKIN DIS(website)
  • PRADER-WILLI SYNDROME (PWS) (website)
  • THERAPEUTICS FOR RARE AND NEGLECTED DISEASES – SCIENCE(website)
  • WIRELESS AT-RISK INFANT MONITORING SYSTEM(website)

2011

  • Drug Repurposing and Rediscovery for Gastrointestinal Stromal Tumors (website)